title: Long-term treatment of somatostatin analog-refractory growth hormone-secreting pituitary tumors with pegvisomant alone or combined with long-acting somatostatin analogs: a retrospective analysis of clinical practice and outcomes.
abstract: BACKGROUND
  Pegvisomant (PEGV) is widely used, alone or with somatostatin analogs (SSA), for GH-secreting pituitary tumors poorly controlled by SSAs alone. No information is available on specific indications for or relative efficacies of PEGV+SSA versus PEGV monotherapy. Aim of our study was to characterize real-life clinical use of PEGV vs. PEGV+SSA for SSA-resistant acromegaly (patient selection, long-term outcomes, adverse event rates, doses required to achieve control).
  
  
  METHODS
  A retrospective analysis of data collected in 2005-2010 in five hospital-based endocrinology centers in Rome was performed. Sixty-two adult acromegaly patients treated ≥6 months with PEGV (Group 1, n=35) or PEGV+SSA (Group 2, n=27) after unsuccessful maximal-dose SSA monotherapy (≥12 months) were enroled. Groups were compared in terms of clinical/biochemical characteristics at diagnosis and before PEGV or PEGV+SSA was started (baseline) and end-of-follow-up outcomes (IGF-I levels, adverse event rates, final PEGV doses).
  
  
  RESULTS
  Group 2 showed higher IGF-I and GH levels and sleep apnea rates, higher rates residual tumor tissue at baseline, more substantial responses to SSA monotherapy and worse outcomes (IGF-I normalization rates, final IGF-I levels). Tumor growth and hepatotoxicity events were rare in both groups. Final daily PEGV doses were similar and significantly increased with treatment duration in both groups.
  
  
  CONCLUSIONS
  PEGV and PEGV+SSA are safe, effective solutions for managing SSA-refractory acromegaly. PEGV+SSA tends to be used for more aggressive disease associated with detectable tumor tissue. With both regimens, ongoing monitoring of responses is important since PEGV doses needed to maintain IGF-I control are likely to increase over time.
authors.affiliation: Department of Endocrinology, Catholic University, School of Medicine, Largo A, Gemelli 8, 00168, Rome, Italy. abianchi69@yahoo.it
authors.forename: Antonio
authors.initials: A
authors.lastname: Bianchi
authors.forename: Ferdinando
authors.initials: F
authors.lastname: Valentini
authors.forename: Raffaella
authors.initials: R
authors.lastname: Iuorio
authors.forename: Maurizio
authors.initials: M
authors.lastname: Poggi
authors.forename: Roberto
authors.initials: R
authors.lastname: Baldelli
authors.forename: Marina
authors.initials: M
authors.lastname: Passeri
authors.forename: Antonella
authors.initials: A
authors.lastname: Giampietro
authors.forename: Linda
authors.initials: L
authors.lastname: Tartaglione
authors.forename: Sabrina
authors.initials: S
authors.lastname: Chiloiro
authors.forename: Marialuisa
authors.initials: M
authors.lastname: Appetecchia
authors.forename: Patrizia
authors.initials: P
authors.lastname: Gargiulo
authors.forename: Andrea
authors.initials: A
authors.lastname: Fabbri
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Toscano
authors.forename: Alfredo
authors.initials: A
authors.lastname: Pontecorvi
authors.forename: Laura
authors.initials: L
authors.lastname: De Marinis
mesh_terms: D000172:Acromegaly / Q000188:drug therapy / Q000209:etiology; D000293:Adolescent; D000328:Adult; D000368:Aged; D018931:Antineoplastic Agents, Hormonal / Q000008:administration & dosage / Q000627:therapeutic use*; D005260:Female; D049912:Growth Hormone-Secreting Pituitary Adenoma / Q000150:complications / Q000188:drug therapy*; D019382:Human Growth Hormone / Q000008:administration & dosage / Q000031:analogs & derivatives* / Q000627:therapeutic use; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D013004:Somatostatin / Q000008:administration & dosage / Q000031:analogs & derivatives / Q000627:therapeutic use*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D018931:Antineoplastic Agents, Hormonal; D019382:Human Growth Hormone; D013004:Somatostatin; C406545:pegvisomant